FDA Grants Mom’s Wish; Gives Orphan Drug Designation

By Amy Dockser Marcus

A mother from Reno, Nev. who was a force behind an application for an orphan drug designation has gotten good news: the FDA approved the request.

Chris Hempel, whose twin daughters have a fatal cholesterol metabolism disorder known as Niemann-Pick Type C (NPC), said that the designation was approved last week. An official at the FDA’s Office of Orphan Products Development confirmed the designation, for a form of a compound called cyclodextrin. The FDA’s move is the first step in the process of developing drugs for rare diseases that affect fewer than 200,000 Americans.

In March, the WSJ reported about a new effort by the FDA to get more applications by holding workshops offering on-the-spot advice. The first one, held in February at the Keck Graduate Institute in California, was attended by Hempel and Ron Browne, a scientist hired by a group of families with children who have NPC. The next one is slated for August, at the University of Minnesota.

At the workshop, Hempel stuck out from a crowd consisting mainly of pharmaceutical and biotech company representatives; the binders that held her application were hot pink, her daughters’ favorite color.

The approval is important for a few reasons. Last year, only 160 applications — out of 250 requests –received an orphan drug designation. So cyclodextrin is now in an elite group. Moreover,”ultra orphan” diseases — affecting fewer than 6,000 patients in the U.S. — such as NPC face particularly steep challenges. These ultra orphans make up less than 15% of orphan designations even though they represent more than 80% of identified rare diseases, according to data prepared by the Kakkis EveryLife Foundation and BioMedical Insights.

Hempel hopes the designation will attract pharmaceutical and biotech companies or investors who might develop cyclodextrin into a drug to help treat NPC. The FDA’s move makes the group eligible to pursue special tax credits that could apply to clinical trials and research. “The designation helps legitimize what we are doing,” says Hempel. “It’s no longer just a mom saying it — the FDA is saying this is a promising compound.”